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Gene Therapy for SPG47 (BFB-101)

This study is testing a gene therapy called BFB-101 (AAV9-CBh-AP4B1) for children with SPG47 (Hereditary Spastic Paraplegia Type 47). SPG47 is a rare genetic condition that causes muscle stiffness (spasticity) and developmental delays. The main goals are to see if BFB-101 is safe and well-tolerated, and if it can improve motor skills and development. You may be able to participate if you are a child between 12 months and 5 years old with a confirmed diagnosis of SPG47. The study plans to enroll 5 participants. The study will track any serious side effects for 60 months (5 years).

Study design
This is an interventional study, meaning participants will receive a treatment. It is a single-center, open-label study, meaning both you and the study team will know what treatment is being given. It plans to enroll 5 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 60 months (5 years) to monitor for unanticipated treatment-related toxicities.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06948019

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

Not Yet Recruiting
PHASE1Ages 12–60InterventionalTreatment
BlackfinBio Ltd
~5 participants
Updated 2025-04-28 on ClinicalTrials.gov
What's tested:BFB-101 (AAV9-CBh-AP4B1)

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG47
Measured over 60 months

Conditions

HSP
Hereditary Spastic Paraplegia
Hereditary Spastic Paraparesis
Hereditary Spastic Paraplegia Type 50
Hereditary Spastic Paraplegia Type 47
Hereditary Spastic Paraplegia Type 51
Hereditary Spastic Paraplegia Type 52
SPG47
AP4B1
Neurogenetic Disorders
Neurodevelopmental Conditions
Movement Disorders
Gene Therapy

Explore related trials

Hereditary Spastic ParaplegiaAP4B1 Gene

NCT06948019

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Boston Children's Hospital

    Boston, Massachusettsstudy coordinator listed

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Darius Ebrahimi-Fakhari, MD, PhD
[email protected]
617-355-0097
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

What this trial measures

  • Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG4760 months

    Incidence of unanticipated treatment-related toxicities, Grade 3 or higher, in participants with SPG47 will be determined from the collection of occurrence and severity of serious adverse events (SAEs). Adverse events will be determined according to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.